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EXCEL Continuing Education.

A Platform for Gene Therapy Delivery: Adeno-Associated Virus (AAV) Vectors

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Overview / Abstract:

Program Overview

The goal of gene therapy is to treat diseases at the genetic level by replacing deficient enzymes in target tissues, therefore, changing the course of the disease. Simply stated, it is the transfer of genetic material for disease therapy or prevention. Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in several trials across target organs, including eye, liver, skeletal muscle, and the central nervous system.

The goal of this CME-certified activity is to increase physicians' knowledge of clinical considerations relating to the use of adeno-associated virus (AAV) vectors in gene therapy, and its impact on the treatment of several rare disorders.

Learning Objectives

Upon completion of this activity, participants should be better able to:

Analyze gene‐based treatment approaches that have the potential to change the disease course for patients with genetic disorders
Review the characteristics of AAV that allow it to be used as a vector for gene therapy
Evaluate evidence for the use of gene therapies, including AAV-based, that are approved or pending approval for the management of rare disorders
Counsel patients about clinical-trial opportunities


Sep 08, 2022


Physician CME



Credits / Hours

0.75 AMA PRA Category 1 Credit(s)™



Presenters / Authors / Faculty


Clinical Geneticist
Underdown/Yeomans Family Endowed Professor in Connective Tissue Disorders Care
Medical Director, Office of Faculty Development
Children’s Mercy Kansas City
Associate Professor of Pediatrics
University of Missouri – Kansas City School of Medicine
Clinical Associate Professor of Internal Medicine
University of Kansas School of Medicine

Sponsors / Supporters / Grant Providers


Keywords / Search Terms

Relias LLC Relias LLC., FreeCME., Gene Therapy, Adeno-Associated Virus FreeCME

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